Gene Therapy Fails to Aid Dystrophy, Fibrosis Patients : SCIENCE FILE: An exploration of issues and trends affecting science, medicine and the environment.

Two widely touted gene therapy experiments--one for muscular dystrophy and the other for cystic fibrosis--have failed to help victims of the two diseases, according to reports in the New England Journal of Medicine. The muscular dystrophy therapy involves the injection of millions of immature muscle cells, called myoblasts, into those who have the disabling disease in hopes that they will fuse with the patient’s cells, inserting a healthy gene. A team from Ohio State University found, however, that the procedure failed to increase strength in the arms of 12 boys who received it.

In a separate report, researchers from the University of North Carolina found that a healthy cystic fibrosis gene contained in a cold virus did not improve breathing in 12 volunteers. An inflammatory response to the virus prevented researchers from administering enough of it to be beneficial.